A Phase 4, Multicenter, Double-blind, Study to Investigate the Efficacy, Safety, and Tolerability of 3 Active Doses of Respreeza® / Zemaira® Weekly Intravenous Infusions Administered Over 3 Years as Longterm Maintenance Therapy in Adult Subjects With Emphysema Related to Alpha1 Antitrypsin Deficiency
试验速览
- 阶段
- 4 期
- 状态
- 尚未招募
- 发起方
- CSL Behring
- 入组人数
- 270
- 主要终点
- Annual rate of change in adjusted lung density
研究概览
简要总结
This is a multicenter, parallel-group, double-blind, randomized phase 4 study designed to identify the optimal dose of CE1226 (2 active doses) to slow disease progression as assessed by reduced rates of annual lung density decline in alpha-1 antitrypsin (AAT) deficient participants over 3 years as compared with the marketed dose 60 milligrams per kilogram (mg/kg).
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
盲法说明
Sponsor will also be blinded to treatment allocation.
入排标准
- 年龄范围
- 18 Years 至 65 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •• Age greater than or equal to (>=) 18 and less than or equal to (<=) 65 years at the time of providing written informed consent.
- •• Confirmed diagnosis of emphysema related to AATD with either the PiZZ, PiZ(null), or Pi(null/null) genotype with documented serum AAT levels less than (<) 11 micrometer (μM) (or < 50 mg/dL [milligram/deciliter]) at any time before the first administration of CE1226 on Day 1 (Baseline).
排除标准
- •• Participants should not have acute illness or pulmonary exacerbation within 6 weeks before the first administration of CE1226 on Day 1 (Baseline).
- •• Participants should not have previously received gene therapy for AATD at any point.
- •• Participants with liver disease secondary to AATD.
研究组 & 干预措施
CE1226 medium dose
CE1226 at medium dose.
干预措施: CE1226 (Biological)
CE1226 high dose
CE1226 at high dose.
干预措施: CE1226 (Biological)
CE1226 low dose
CE1226 at low dose.
干预措施: CE1226 (Biological)
结局指标
主要结局
Annual rate of change in adjusted lung density
时间窗: From Baseline to Month 36
The annual rate of change (expressed as gram per liter per year \[g/L/year\]) in adjusted lung density (15th percentile lung density \[PD15\]) will be calculated on CT scan assessments of whole lung at total lung capacity (TLC).
次要结局
- Annual rate of change in forced expiratory volume in 1 second percent predicted (FEV1%)(From Baseline to Month 36)
- Annual rate of change in diffusion capacity of carbon monoxide (DLco)(From Baseline to Month 36)
- Number of severe pulmonary exacerbations(From screening up to Month 36)
- Duration of severe pulmonary exacerbations(From screening up to Month 36)
- Number of participants experiencing treatment emergent adverse events (TEAEs)(From screening up to Month 36)
- Percentage of participants experiencing TEAEs(From screening up to Month 36)